Biopharmaceutical News #28
Author: Jean-Claude Muller, 穆卓Executive Editor at BtoBioInnovation, jcm@btobioinnovation.com
Drugs at clinical stage
The US FDA Oncologic Drugs Advisory Committee (ODAC), with a vote 10-0, recommends the approval of Novartis' CTL019 or tisagentlecleucel, an investigational chimeric antigen receptor T cell (CAR-T) therapy as a treatment of relapsed or refractory (r:r) pediatric and yound adult patients with B-cell acute lymphoblastic leukemia (ALL).
If approved, later this year, the drug will be the first CAR-T cell therapy availablei in the world. CTL019 previously received US FDA Breakthrough Therapy designation and is still under Priority Review by the FDA
Acute lymphoblastic leukemia comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the USA. Effective treatment options for patients with r/r ALL are limited. In pediatric and young adult patients with B-cell ALL that have relapsed multiple times or become refractory to treatment, the five-year disease-free survival is less than 10-30%.
The highly personalized treatment CAR T-cell therapy is a type of cancer immunotherapyor that harnesses the body's immune system to take on cancer cells. CAR-T is manufactured for each individual patient using their own cells. During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the manufacturing facility to create T cells that are genetically coded to express a chimeric antigen receptor to recognize and fight cancer cells and other B-cells expressing a specific antigen.
CTL019 was first developed by the University of Pennsylvania (Penn) and uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular responses as well as persistence of CTL019 after it is infused into the patient, which may be associated with long-lasting remissions in patients. In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Children's Hospital of Philadelphia was the first institution to investigate CTL019 in the treatment of pediatric patients and led the single site trial.
Other late stage CAR-T cell treatments are developed by Kite Pharma, Juno Therapeutics, Bluebird bio in collaboration with Celgene and Cellectis in collaboration with Servier and Pfizer.
This document has been prepared by btobioinnovation and is provided to you for information purposes only. The information contained in this document has been obtained from sources that btobioinnovation believes are reliable but btobioinnovation does not warrant that it is accurate or complete. The views presented in this document are those of btobioinnovation’s editor at the time of writing and are subject to change. btobioinnovation has no obligation to update its opinions or the information in this document.
Last News
- Note de réflexion de fin d’année
- EMA re-examines its opinion and approves Leqembi for the treatment of Alzheimer’s disease
- Le déclassement de la France
Events
News archives
- December 2024
- November 2024
- October 2024
- September 2024
- August 2024
- July 2024
- June 2024
- March 2024
- February 2024
- January 2024
- November 2023
- September 2023
- July 2023
- April 2023
- March 2023
- January 2023
- December 2022
- November 2022
- October 2022
- August 2022
- June 2022
- May 2022
- April 2022
- March 2022
- January 2022
- December 2021
- November 2021
- October 2021
- September 2021
- August 2021
- July 2021
- June 2021
- May 2021
- April 2021
- March 2021
- February 2021
- January 2021
- December 2020
- November 2020
- October 2020
- September 2020
- July 2020
- June 2020
- May 2020
- April 2020
- March 2020
- February 2020
- January 2020
- December 2019
- November 2019
- October 2019
- September 2019
- June 2019
- May 2019
- April 2019
- March 2019
- February 2019
- January 2019
- December 2018
- October 2018
- June 2018
- May 2018
- March 2018
- February 2018
- January 2018
- December 2017
- November 2017
- October 2017
- September 2017
- August 2017
- July 2017
- June 2017
- May 2017
- April 2017
- March 2017
- February 2017
- January 2017
- December 2016
- November 2016
- September 2016
- July 2016
- June 2016
- May 2016
- April 2016
- March 2016
- February 2016
- January 2016
- December 2015
- November 2015
- October 2015
- September 2015
- August 2015
- July 2015
- June 2015
- May 2015
- April 2015
- March 2015
- February 2015
- January 2015
- December 2014
- November 2014
- October 2014
- September 2014
- June 2014
- May 2014
- April 2014
- March 2014
- January 2014
- November 2013
- September 2013
- July 2013
- May 2013
- April 2013
- March 2013
- January 2013
- December 2012
- November 2012
- October 2012
- March 2012